Safety and efficacy of BAY 81-8973 for surgery in previously treated patients with haemophilia A: results of the LEOPOLD clinical trial programme.

Abstract

INTRODUCTION:

BAY 81-8973 is a recombinant factor VIII (rFVIII) with the same amino acid sequence as Bayer's sucrose-formulated rFVIII (rFVIII-FS) but manufactured with certain more advanced technologies.

AIM:

To describe surgery outcomes with BAY 81-8973 in the LEOPOLD trials.

METHODS:

Male patients with severe haemophilia A and no inhibitors aged 12-65 years with ≥150 exposure days (EDs) to FVIII (LEOPOLD I and II), or aged ≤12 years with ≥50 EDs to FVIII (LEOPOLD Kids), received BAY 81-8973 based on dosing recommendations for rFVIII-FS according to surgical requirements. Haemostasis-related complications, investigator/surgeon assessment of haemostasis, blood loss, need for transfusion and use of BAY 81-8973 were determined.

RESULTS:

In LEOPOLD I and II, 11 patients (mean age, 35.3 years) underwent 13 major surgeries. In LEOPOLD Kids, one patient (aged 6 years) underwent one major surgery. Thirty-two adult and paediatric patients underwent 46 minor surgeries. Haemostasis was rated good or excellent in all major and minor surgeries. Blood loss during surgery did not exceed expected amounts; blood transfusions were required in three of the 14 major surgeries. For major surgeries in LEOPOLD I and II, patients received a presurgical 50-IU kg(-1) dose of BAY 81-8973; median nominal dose on day of surgery was 7000 IU (107.5 IU kg(-1) ). Total BAY 81-8973 dose was 2500 IU (108.7 IU kg(-1) ) on the day of the only major surgery in LEOPOLD Kids. No haemostasis-related complications were reported.

CONCLUSIONS:

Haemostatic control with BAY 81-8973 during all surgeries in the LEOPOLD trials was good or excellent, with no haemostasis-related complications.

• Authors: J Oldenburg, J Windyga, K Hampton, S Lalezari, D Tseneklidou-Stoeter, H Beckmann, M Maas Enriquez
• Journal: Haemophilia : the official journal of the World Federation of Hemophilia (Haemophilia) Vol. 22 Issue 3 Pg. 349-53 (May 2016) ISSN: 1365-2516 [Electronic] England
• PMID: 26931631 (Publication Type: Clinical Trial, Phase I, Clinical Trial, Phase II, Clinical Trial, Phase III, Journal Article, Multicenter Study)
• Copyright: © 2016 John Wiley & Sons Ltd.

Chemical References

• Coagulants
• F8 protein, human
• Factor VIII

Topics

• Adolescent
• Adult
• Aged
• Blood Transfusion
• Child
• Coagulants (adverse effects, pharmacokinetics, therapeutic use)
• Cross-Over Studies
• Factor VIII (adverse effects, pharmacokinetics, therapeutic use)
• Hemophilia A (drug therapy, pathology)
• Hemorrhage (prevention & control)
• Humans
• Male
• Middle Aged
• Surgical Procedures, Operative
• Treatment Outcome
• Young Adult